Company Successfully Transitions into Late-Stage Development with Amigal for Fabry Disease Entering Phase 3
CRANBURY, N.J., Aug. 5 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced financial results for the second quarter 2009.
On a reported basis calculated in accordance with U.S. Generally Accepted Accounting Principles (GAAP), Amicus announced a net loss attributable to common stockholders of $0.60 per share ($0.51 per share on a non-GAAP basis) for the three months ended June 30, 2009. As of June 30, 2009, cash, cash equivalents, and marketable securities totaled $100.4 million.
John F. Crowley, President and CEO of Amicus stated, "The second quarter was marked by a significant achievement for Amicus with our lead program, Amigal for Fabry disease, entering Phase 3 development." Crowley continued, "This is a major step for the Company as we have now transitioned to a late-stage biopharmaceutical company and are one step closer to bringing to market an important, novel treatment to patients who suffer with Fabry disease."
Clinical Program Updates
Amigal(TM) (migalastat hydrochloride) for the treatment of Fabry disease
In the second quarter of 2009, Amicus announced that the Company reached agreement with the U.S. Food and Drug Administration (FDA) on the key protocol design elements of its pivotal trial, including the use of the surrogate primary endpoint of the change in the amount of kidney interstitial capillary GL-3, the substrate that accumulates in the cells of Fabry patients. In addition, the FDA is in agreement that the Company is eligible to seek Accelerated Approval for Amigal according to Subpart H regulations. Amicus has begun submitting the Phase 3 protocol to investigational sites worldwide and expects to begin the dosing of subjects in the second half of this year.
As previously announced, the Phase 3 trial will evaluate the efficacy, safety, and pharmacodynamics of Amigal in males and females with Fabry disease. The trial will consist of a six-month double-blind, randomized, placebo-controlled treatment stage and will enroll approximately 60 subjects who are naive to enzyme replacement therapy (ERT) or who have not received ERT for at least six months prior to the start of treatment with Amigal. The Amigal treatment arm dose and regimen will be 150 mg every other day.
The primary endpoint will be the change in the amount of kidney interstitial capillary GL-3 as measured in kidney biopsies using histology. Secondary endpoints will include safety and tolerability, kidney GL-3 as measured in urine, and an assessment of renal function (including glomerular filtration rate (GFR) and 24-hour urine protein).
Additionally, the FDA and Amicus reached agreement on an improved methodology for the histological evaluation of GL-3 in the kidney biopsies. An analysis of the Phase 2 and Phase 2 extension study biopsies using the improved methodology demonstrated that all of the evaluable baseline samples had detectable interstitial capillary GL-3 and that reductions were observed in eight of the nine responders.
It is expected that approximately 30 clinical sites worldwide will participate in this trial.
Plicera(TM) (afegostat tartrate) for the treatment of Gaucher Disease
A Phase 2 clinical trial of the Company's investigational drug Plicera is ongoing. This 6-month study is designed to evaluate safety, dosing, and preliminary efficacy, as measured by the standard endpoints in Gaucher disease. The Company expects to report the results early in the fourth quarter of 2009.
Amicus continues to work closely with its partner, Shire HGT, to prepare for Phase 3 development of Plicera pending the results of the ongoing Phase 2 trial.
AT2220 (1-deoxynojirimycin HCl) for the treatment of Pompe Disease
As previously reported, the Company suspended enrollment for the Phase 2 clinical trial of its investigational drug AT2220 for the treatment of Pompe Disease and received notice from the U.S. Food and Drug Administration (FDA) that the trial is on clinical hold.
The Company continues to work closely with the FDA to determine appropriate next steps for advancing the development of AT2220 and expects to provide guidance on this progress over the upcoming months.
Additionally, Amicus continues to be encouraged with its preclinical studies with AT2220 in combination with ERT. As previously announced, the Company expects to report additional data from these activities throughout the remainder of 2009.
Preclinical Chaperone Programs
Amicus continues to invest in research to assess the potential for applying its versatile chaperone technology platform to the treatment of a broad range of human genetic diseases. As part of this effort, Amicus continues to conduct preclinical studies in Parkinson's disease and is investing in new research aimed at evaluating disease targets for other neurodegenerative and genetic disorders.
Appointment of New Director
The Company announced today the election of James Barrett, Ph.D., to its Board of Directors. Dr. Barrett currently serves as General Partner of New Enterprise Associates (NEA), the Company's single largest shareholder, where he specializes in biotechnology and works with members of NEA's healthcare investment group on medical devices, healthcare information systems and healthcare services companies. Prior to joining NEA in 2001, Dr. Barrett served as Founder, Chairman and CEO of Sensors for Medicine and Science (1997 - 2001) where he remains Chairman. Prior to that, he led three NEA-funded companies, serving as Chairman and CEO of Genetic Therapy, Inc. (1987 - 1995), President and CEO of Life Technologies (1985 - 1987), and President and CEO of Bethesda Research Labs (1982 - 1983). He currently serves on the Board of Directors for several life sciences companies including Inhibitex, Inc. and Targacept, Inc.
Dr. Barrett received a Ph.D. in Biochemistry at the University of Tennessee, his MBA from the University of Santa Clara, and a BS in Chemistry from Boston College.
Shire HGT Collaboration
In November 2007, Amicus entered into a strategic collaboration with Shire Human Genetic Therapies, Inc., a wholly-owned subsidiary of Shire plc, to jointly develop Amicus' three lead pharmacological chaperone compounds for lysosomal storage disorders: Amigal, Plicera and AT2220. Under the terms of the collaboration, Shire will pay development and sales milestones up to a maximum of $390 million, and will also pay tiered, double-digit royalties on net sales of the products. Shire also reimburses worldwide development costs on a 50/50 basis, and in return Shire received rights to commercialize these products outside of the U.S. while Amicus retains all rights to commercialize these products in the U.S. In addition, Amicus leads development operations through the end of Phase 2 clinical trials. The companies then share responsibility for Phase 3 clinical trial development leveraging Shire's significant ex-U.S.
